The Catalyst

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While the number of patients with any individual rare disease is relatively small, they collectively affect 1 in 10 Americans. The community successfully comes together to raise awareness on international Rare Disease Day every February, but the challenge is how to maintain the momentum – and remain united - for the remaining 364 days of the year.

In January, the Centers for Medicaid and Medicare Services (CMS) issued a proposed rule that would have adversely altered Medicare Part D’s six protected classes for prescription medication, restricting Medicare beneficiary access to antidepressants and immunosuppressants beginning in 2015.

The development of new medical breakthroughs depends on the patients who volunteer to participate in clinical trials, but according to the Center for Information and Study on Clinical Research Participation, only 6 percent of clinical trials are completed on time and half of clinical research sites enroll one or no patients in their study.

The recent spotlight on prescription drug abuse has overshadowed an important patient population that must deal with chronic pain everyday. Addicts, overdoses and mentions of the “opioid epidemic” in the media has made is increasingly difficult for the people who depend on these medicines to be able to get them.

Each patient is unique, which means that medicines and therapies often need to be tailored to fit individual needs to ensure the best possible care. While the research and development (R&D) pipeline holds the promise of new, innovative treatments for those suffering from a variety of diseases, it is important to explore all options to improve patient health.

A patient should never have to worry about the safety of their medicines. Unfortunately, patient’s lives are put at risk across the country due to counterfeit, substandard, or otherwise unsafe medicines.

Since taking effect in 2006, Medicare Part D has provided seniors with critical access to the prescription medications they need. As part of the program, patients have broader access to medicines that fall into one of six “protected classes” that include anticonvulsants, antidepressants, antineoplastics, antipsychotics, antiretrovirals and immunosuppressants.

Since the Accelerating Medicines Partnership (AMP) was announced last month, there’s been robust discussion throughout the scientific community and the general public about the goals of this unique partnership between the National Institutes of Health (NIH), several non-profit disease foundations, 10 biopharmaceutical compani

We revisit the research and development (R&D) pipeline quite a bit, because it is a crucially important aspect of modern medicine. Having recently released our Medicines in Development report on the 180 medicines in the pipeline to treat diabetes and related conditions, it is important to recognize the steps that fuel innovation and medical advances.